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Breakthrough for Patients

By: Dr. Arie Belldegrun

Perhaps the best line in the history of scientific literature comes toward the end of the famous 1953 Nature paper by James Watson and Francis Crick describing the structure of DNA, the building blocks of life.

“It has not escaped our notice,” they wrote, “that the specific pairing we have postulated immediately suggests a possible copying mechanism for the genetic material.”

That “copying mechanism for the genetic material” gave rise to much of modern biology. Watson and Crick’s words have now been called a “classic understatement” and “it has not escaped our notice” has, in science, become something of an inside joke among academics. 

But there is another reason why Watson and Crick may have been coy about the specifics of their great discovery: the details were still being worked out, and science does not always deliver the outcome we expect. What may seem now to be an understatement may have been, at the beginning of the genomic age, simply implied caution.

While I can’t claim to have stood on the precipice of a revolution in biology anything like that of Watson, Crick, and Rosalind Franklin, I have had the experience of seeing a new technology born and wondering whether the enormous promise of test-tube experiments could see its potential realized.

The technology I saw in its infancy following my time as a fellow at the National Cancer Institute (NCI) under my mentor, Dr. Steve Rosenberg, was the use of T cells to fight cancer that led to the birth of CAR-T therapy. This innovation takes the T-cell – a workhorse of the immune system – and pairs it with cell receptors that are engineered to recognize cancer cells. To borrow from Watson and Crick, it did not escape my notice that this approach could provide a powerful new tool to fight cancer. But I was under no illusion that the road would be easy.

CAR-T is an elegant technology, and a tricky one, requiring each treatment to be specifically tailored to a specific patient. But the promise was so overwhelming that I, along with some of my closest friends, bet our own resources on bringing the idea to patients.

Now, we are on the verge of seeing that groundbreaking technology become a potential reality for more than just a select few patients. Both Kite and Novartis have asked the U.S. Food and Drug Administration to approve CAR-T therapies, and the number of successful clinical trials is growing by the month. Where once we spoke in cautious terms about “possible” benefits, we are now increasingly confident. That doesn’t mean we are not continuing to push forward, refining the technology, streamlining the logistics of a personalized therapy, discovering new innovations that allow us to mitigate adverse events, and bringing this approach to new types of cancer.

I expect 2017 will be a year of revolutions and Kite is excited to play its part. While the road is still long, the potential is worth the journey. In the weeks and months to come, I plan to weigh in on what is happening in the cell therapy space, what that means for science and, most importantly, what it means for patients.